Suramin for Autism?


SAN DIEGO, Calif. (Ivanhoe Newswire) — The CDC estimates that one in 68 children in the U.S. are on the autism spectrum. Right now, there are no FDA approved treatments for the disorder or its symptoms, which can include difficulty with social skills, communication and repetitive behavior. That’s why results of a small clinical trial in San Diego have grabbed the attention of researchers and families.

Robert K. Naviaux, MD, PhD, Professor of Genetics at the Departments of Medicine, Pediatrics, and Pathology, and Co-director of the Mitochondrial and Metabolic Disease Center at UC San Diego School of Medicine suspected the cause of autism might be metabolic dysfunction, where the energy molecule ATP is “outside” cells. He researched more than two thousand drugs and found one that might help. That drug was Suramin. Dr. Naviaux tested one dose in a clinical trial of ten boys. Five got the drug.

Dr. Naviaux  shared, “Children began to talk sometimes for the first time in sentences in their life.” (Read Full Interview)

Boys who got Suramin had autism severity scores drop from eight-point-six to seven, the lowest point on the spectrum. They improved social, language and fine motor skills, and found relief from repetitive motions and fragmented sleep. Miles McInerney was in the trial but did not receive Suramin; he still wanted to help.

“I should generally be interested in the ability to possibly find a way that people with worse autism or struggle more with autism than I do, to possibly be able to find a solution that can help them better communicate,” said McInerney.

He now uses rowing to reduce the stress related to his autism. For those who did get the drug, Dr. Naviaux says most but not all the effects wore off in eight weeks.

“Some children had learned to tie their shoes for the first time, and other children had learned to zip up a jacket. Those fine motors skills were motor memory that had been retained.” Dr. Naviaux continued.

McInerney and his mom are encouraged by the results.

Dr. Naviaux says next there will be several phase two trials to determine safety and efficacy for Suramin. He suspects it will be three to five years before phase three trials begin.

Contributors to this news report include: Wendy Chioji, Field Producer; Rusty Reed, Videographer; Cyndy McGrath, Supervising Producer; Hayley Hudson, Assistant Producer; Roque Correa, Editor.

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REPORT:        MB #4416


AUTISM: Autism, or autism spectrum disorder, refers to a range of conditions characterized by challenges with social skills, repetitive behaviors, speech and nonverbal communication, as well as by unique strengths and differences. We now know that there is not one autism but many types, caused by different combinations of genetic and environmental influences. The term “spectrum” reflects the wide variation in challenges and strengths possessed by each person with autism. Autism’s most-obvious signs tend to appear between two and three years of age. In some cases, it can be diagnosed as early as 18 months. Some developmental delays associated with autism can be identified and addressed even earlier. The CDC estimates autism’s prevalence as one in 68 children in the United States. This includes one in 42 boys and one in 189 girls.



BACKGROUND: African Trypanosomiasis, also known as “sleeping sickness,” is caused by microscopic parasites. It is transmitted by the tsetse fly, which is found only in rural Africa. Although the infection is not found in the United States, historically, it has been a serious public health problem in some regions of sub-Saharan Africa. Currently, about 10,000 new cases each year are reported to the World Health organization; however, it is believed that many cases go undiagnosed and unreported. Sleeping sickness is curable with medication, but is fatal if left untreated. For the past 50 years the manufacturer of the drug Suramin has been making a few kilograms of it and providing it to the World Health Organization in Geneva to be distributed in Africa to treat African sleeping sickness. But Dr. Robert Naviaux, MD, PhD, at UC San Diego School of Medicine thought Suramin might have another use.

(Source: & Robert Naviaux, MD, PhD)


SURAMIN: Dr. Naviaux began by testing Suramin on mouse models, he explained, “We found that Suramin corrected all of the behavioral and learning disabilities if you started in the first month of life. We did another study where we looked at animals with autism symptoms that were the age equivalent of a 30-year old that had never been treated before. And even in that animal the behavioral symptoms and social abnormalities were completely corrected.” Researchers moved on to a trial of ten children, and saw positive results in those who received Suramin. The next step is a series of new clinical trials culminating in a Phase III multicenter trial that could be conducted over the next three to five years.

(Source: Robert Naviaux, MD, PhD)




Scott Lafee, Media Relations


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Doctor Q and A

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